PB2022: ELTROMBOPAG IN MYELODYSPLASTIC SYNDROME AND CHRONIC MYELOMONOCYTIC LEUKEMIA: A SINGLE-CENTER EXPERIENCE
Authors: Amaia Lopez Peña, Marta Sofia Ordas Miguelez, Carmen Rodriguez Lefler, Pablo Estuardo Lopez Gomez, Mar Herrero Gutierrez, Laura Fernandez Cuezva, Jhossyuriel Obregon Membreño, Rita Gonzalez Resina, Laura Villarroya Martínez, Leire Etxebarria Bahillo, Rafael Monleon Gil, Ana Cristina Godoy Molias, Miguel Diaz Morfa, Isabel Izquierdo Garcia, Pilar Delgado Beltran, Gonzalo Caballero Navarro
Journal: HemaSphere (2023)

Topic: 10. Myelodysplastic syndromes - Clinical Background: Thrombocytopenia and platelet dysfunction in myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML) contribute to hemorrhagic complications, occasionally life-threatening. Eltrombopag (EPAG), a thrombopoietin receptor agonist, stimulates megakaryocyte differentiation and proliferation. In some studies, efficacy of EPAG in MDS with thrombocytopenia has been proved. Aims: To evaluate the effect of EPAG in platelet count, transfusion dependence and number of bleeding episodes in patients with MDS and CMML. Methods: Retrospective observational study based on patients diagnosed with MDS or CMML with thrombocytopenia from January 2012 to February 2023 treated with EPAG. Diagnosis was assessed by 2016 WHO classification and risk category by Revised International Prognostic Scoring System (IPSS-R) for MDS and CMML-specific Prognostic Scoring System (CPSS) for CMML. Response to treatment was defined as an increase in platelets greater than 20% compared to baseline count or more than 20 x103/µL platelets compared to the previous count. Results: We studied 14 patients, 10 diagnosed with MDS and 4 with CMML. Characteristics of our cohort are described in table 1. The majority of patients with MDS (8/10) were low or very low risk and all of the CMML patients were intermediate risk. The median platelet count at diagnosis was 47 x103/µL (35.5-80.5). Prior to EPAG, half of the patients (7/14) had bleeding history and half of the patients (7/14) were dependent on platelet transfusions. The median platelet count at the start of EPAG was 18 x103/µL (10.5-24.3). Eight patients began EPAG at 25 mg/day, three at 50 mg/day and one at 75 mg/day. Doses were adjusted depending on platelet count until either response or withdrawal. The median of treatment was 8 months (3.4-41.5). Eight out of 14 patients (64.3%) responded to treatment after one month, six diagnosed with MDS and two with CMML. Three patients discontinued EPAG before completing three months due to absence of response (2/3) and liver toxicity (1/3). At 3 and 6 months of follow-up six patients (6/11) continued responding. After 3 years of monitoring, five patients (5/11) continued taking EPAG with a good response. Of the remaining patients (6/11), five discontinued treatment due to no response and one has not yet reached that follow-up time. The patient with the longest follow-up time (6 years) remains responding to EPAG (platelets >100 x103/µL). Of the patients with transfusion dependence (7/14), three responded to EPAG and achieved transfusion independence. Of the patients with bleeding history (7/14), three responded to EPAG but only one stopped bleeding. During treatment, eight patients had hyperbilirubinemia and three hypertransaminasemia but only two required suspension of EPAG. No patients suffered from thrombosis or progression to acute myeloid leukemia. The diagnosis of five of the patients (5/6) that responded at 3-6 months was MDS and one (1/6) CMML. Summary/Conclusion: Our study showed that EPAG was effective in increasing the platelet count in 42.8% of the patients after 3-6 months of treatment. Most of the patients that responded were low risk MDS, taking into account that these patients were the majority of our sample. The use of EPAG and other TPO-RAs in low risk MDS and CMML can be considered in patients with severe thrombocytopenia and haemorrhage, due to the limited available treatments. We consider that further research is needed to establish the effectiveness and safety of this approach.Keywords: CMML, Thrombocytopenia, MDS, Thrombopoietin (TPO)